What are lysosomal storage diseases?

Lysosomal storage diseases are inherited genetic defects that result in an enzyme deficiency, which prevents cells from performing their natural recycling function. This leads to a variety of progressive physical and/or mental deterioration. Approximately 180 New Zealand families are currently affected. Find out more.

About LDNZ

Lysosomal Diseases NZ is a charitable trust dedicated to improve contact between families affected by lysosomal diseases within New Zealand, and supporting research into the causes and treatment of lysosomal diseases and improvements in the clinical care of affected people. Read more about our goals and activities.

Campaign for access to therapies for our diseases

We have been campaigning hard since the early 2000s to get access to specialised therapies for our diseases. Some progress has been achieved but there is still a long way to go. We have made numerous submissions, organised seminars, and taken delegations to officials, politicians and to Pharmac itself.
Our campaign included a complaint to the Office of the Ombudsman, an officer of Parliament charged with scrutiny of the way government agencies administer policy, plus investigations of possible legal action. We are also working with political parties to achieve better systems for decisions about specialised
medicines. Read more...

News

April 2017 newsletter

Pharma Company gives up on Pharmac

After many years of advocacy and Genzyme trying to work with Pharmac to fund Myozyme for our NZ patients with Pompe disease, the company has finally given up and is compassionately funding four Pompe patients. Read our press release.

13th December 2016 Petition Delivered to Government

Samantha Lenik and patients with Pompe and Fabry disease and their supporters delivered the petition to the government yesterday requesting that the government look under urgency to ensure Pharmac has enough funds to properly deliver on the rare disease drug policy. Read more here.